'all we care about': surrey family fights on to fund gene editing procedure for son's rare disease
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the gill family is fighting for their three-year-old son, gurmoh, to receive a gene therapy for a rare disease that threatens his ability to walk.
navpreet gill
a surrey family is racing against time to raise enough money for a gene-editing procedure that could save their three-year-old son’s ability to walk.
when gurmoh was struggling to walk on his own at 1.5 years of age, his parents stalin and navpreet gill knew something was wrong.
the parents were initially told the issue would resolve itself, but gurmoh’s capabilities have only got worse.
earlier this year, gurmoh was diagnosed with a rare, progressive neurodegenerative disease called hereditary spastic paraplegia type 4, which causes weakness and stiffness in the legs.
“this is the only thing we have on our mind, it’s all we do, all we care about,” stalin said.
gurmoh underwent an mri and blood testing, which showed no abnormalities, the father said.
he was first diagnosed with cerebral palsy after his legs completely gave out and he was rushed to b.c. children’s hospital. the family didn’t get the right answer until april.
since then, the family has been searching for treatment, as there is no known cure for the condition.
“my wife sent about 2,000 emails around the globe to all the researchers that were doing any sort of research on this disease,” stalin said.
a doctor from mcgill university, dr. ziv gan-or, responded and said he could develop a personalized gene therapy for gurmoh.
“hsp is a rare disease found in about five out of 100,000 people. that means in canada, there’s about 2,000 patients with this disease,” gan-or said in a statement.
the form of hsp gurmoh has, type 4, is the most common form of the disease, the doctor added.
“he is expected, as this disease progresses, to lose his ability to walk, his ability to talk, his ability to connect with people and he would be completely dependent on his environment and his caretakers,” gan-or said.
the doctor told the gill family that the treatment would cost up to $8 million. the gills reached out to the b.c. and federal governments, thinking they could receive some coverage.
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“we were really happy, like someone is ready to make the therapy medicine within canada,” stalin said.
gurmoh gill, 3, has a rare progressive neurodegenerative disease that will take away his ability to walk, talk and more if no treatment is provided.
sobia moman
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navpreet gill
the provincial government informed the family it could not cover the treatment, so the family started fundraising. in total, $2.4 million has been raised, nearly reaching the initial $2.7 million goal to just kickstart the development of the gene therapy for gurmoh.
along with supporters, the gills walked 120 kilometres from vancouver to the b.c. legislature in victoria from may 21 to 25. last month, the family went to ottawa and stood outside parliament for three days until minister of health marjorie michel met with them and said she would reach out to health canada.
“with this treatment, you edit the gene so it stops making the wrong protein,” stalin said. “ideally, they use one injection and that would be for life to fix everything.”
currently, stalin said the family travels to toronto every 90 days for gurmoh to undergo intensive physical therapy that he says is not available in b.c.
“on average, every month we spend $6,000 to $7,000 on his therapy, but we can afford it. imagine who can’t afford it,” he said.
gurmoh is able to walk right now, but his ability worsens as time progresses. at one point, he could walk up stairs on his own, but he now needs to hold on to someone or something for support, the father explained.
gan-or told the family that gurmoh is in a good spot for the treatment right now since he is still able to walk around, stalin said. if the funding is not secured before his walking capability worsens, the gene therapy may not be as effective, he added.
if gurmoh is able to get the treatment, it would be done by gan-or and other experts who performed gene editing for a child with a different disease last year, gan-or said.
“this approach can be applied for gurmoh as well,” he added.
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“having the technology to correct genetic mutations that cause rare diseases provides hope to millions of canadians living with these disorders, as well as many more millions around the world.”
since making their fight public, the family has been supported by medical institutions, including harvard medical school and boston children’s hospital.
families from all over have also reached out, stalin said.
“a lot of them don’t speak english so they’re asking us for help, hoping that this goes through, the gene therapy can be developed so their own kids with the rare diseases can be helped.” stalin said. “we started it as a fight for our son, but it’s more than just gurmoh.”
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